Tissue replacement in RPE related dystrophies

Prior treatment

• Prior treatment with a gene or cell therapy product
• Patients treated with Luxturna®
• Systemic corticosteroid therapy or other immunosuppressive/ immunomodulating or anti-retroviral drugs within 2 months prior to baseline

Other illness

• History of allergy or sensitivity to one of the products used during the study
• Patients with preformed anti-HLA antibodies against the human Embryonic Stem Cell (hESC) line RC9 with MFI > 2000 or patients with intermediate MFI values (between 500 and 2000) at screening associated to medical history of clinical significance, according to expert’s opinion
• Presence of any ocular disease or ocular media opacity which in the opinion of the investigator precludes accurate evaluation

• Documented diagnosis of retinis pigmentosa based on a genetic test confirming the presence of a monogenic mutation that affects a gene involved in the visual signalling process specifically at the level of RPEs, namely RPE65 or LRAT, or MerTK
• 18 years old ≤ Age ≤ 65 years old
• During the SARS-Cov-2 pandemic period, patient negative to SARS-CoV-2 (PCR or any new test validated)
• For patient of the first cohort: Visual acuity ≤ 20/200 in the best eye (legally blind). For patient of the second cohort: 20/63 > Visual acuity > 20/800 in the worst eye And Visual field exceeding 10° central to the V4 test in the worst eye And Visible photoreceptor outer nuclear layer (ONL) on a spectral domain optical coherence tomography (OCT) scan